Single intravenous (iv) dose of human low density lipoprotein receptor (ldlr) gene therapy (DrugBank: -)
1 disease| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 79 | 家族性高コレステロール血症（ホモ接合体） | 1 |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04080050 (ClinicalTrials.gov) | September 30, 2019 | 3/9/2019 | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 | Homozygous Familial Hypercholesterolemia (HoFH) | Drug: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy | Regenxbio Inc. | NULL | Enrolling by invitation | 18 Years | N/A | All | 12 | United States;Canada;Italy;Netherlands |